A Phase 3 trial of intrathecal onasemnogene abeparvovec (OAV101 IT) – an investigational gene replacement therapy – in children ages two to 18 years with spinal muscular atrophy (SMA) has shown positive results.
OAV101 IT is designed to directly address the genetic root cause of the disease by replacing the nonworking SMN1 gene with a single dose.
Pharmaceutical company Novartis, which has developed the treatment, announced positive safety and efficacy results from the Phase III STEER study, which showed that treatment with OAV101 IT led to a statistically significant improvement of motor ability and disease progression.
In the Phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam also demonstrated stabilisation of motor function over 52 weeks of follow-up.
“In the STEER study evaluating treatment-naïve patients, OAV101 IT demonstrated a statistically significant improvement in motor function across a broad SMA population,” said Crystal Proud, paediatric neurologist and a principal investigator at Children’s Hospital of the King’s Daughters.
“These results – paired with those in the STRENGTH study – support the potential for OAV101 IT to be a meaningful treatment option for people living with SMA with a goal of maintaining or improving motor function through a one-time therapy.”
“The data presented today from our OAV101 IT program reinforce our belief in this therapy, which has the potential to have a meaningful impact on a broad range of people with SMA through its continuous benefit via a one-time dose,” said Shreeram Aradhye, president, development and chief medical officer, Novartis.
“Together with patients, caregivers and healthcare professionals, we are committed to continuing to advance our mission to lead innovation in SMA treatment and broaden therapy options with our gene replacement therapies.”
